日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Inhaled DNAI1 mRNA therapy for treatment of primary ciliary dyskinesia.

吸入式 DNAI1 mRNA 疗法治疗原发性纤毛运动障碍

Hennig Mirko, Bhattacharjee Rumpa B, Agarwal Ishita, Alfaifi Ali, Casillas Jade E, Chavez Sofia, Ishimaru Daniella, Liston David, Mohapatra Sakya, Molla Touhidul, Pathare Suyog, Sidhu Maninder S, Wang Peng, Wang Zechen, Lombana T Noelle, Kharitonov Vladimir G, Couch Jessica A, Lockhart David J, Wustman Brandon A

Lipid nanoparticle-encapsulated Dnai1 mRNA rescues ciliary activity in primary ciliary dyskinesia mouse cell models

脂质纳米颗粒包裹的Dnai1 mRNA可挽救原发性纤毛运动障碍小鼠细胞模型中的纤毛活动。

Smith, Amanda J; Sears, Patrick R; Hennig, Mirko; Bhattacharjee, Rumpa B; Yin, Weining; Golliher, Hannah; Ishimaru, Daniella; Lombana, T Noelle; Lockhart, David J; Wustman, Brandon A; Ostrowski, Lawrence E

In vivo editing of lung stem cells for durable gene correction in mice

利用小鼠肺干细胞进行体内编辑以实现持久基因校正

Sun, Yehui; Chatterjee, Sumanta; Lian, Xizhen; Traylor, Zachary; Sattiraju, Sandhya R; Xiao, Yufen; Dilliard, Sean A; Sung, Yun-Chieh; Kim, Minjeong; Lee, Sang M; Moore, Stephen; Wang, Xu; Zhang, Di; Wu, Shiying; Basak, Pratima; Wang, Jialu; Liu, Jing; Mann, Rachel J; LePage, David F; Jiang, Weihong; Abid, Shadaan; Hennig, Mirko; Martinez, Anna; Wustman, Brandon A; Lockhart, David J; Jain, Raksha; Conlon, Ronald A; Drumm, Mitchell L; Hodges, Craig A; Siegwart, Daniel J

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

当与输注阿加糖酶联合使用时,口服米格司他盐酸盐可提高法布里病患者体内活性α-半乳糖苷酶A的全身暴露量和组织水平。

Warnock, David G; Bichet, Daniel G; Holida, Myrl; Goker-Alpan, Ozlem; Nicholls, Kathy; Thomas, Mark; Eyskens, Francois; Shankar, Suma; Adera, Mathews; Sitaraman, Sheela; Khanna, Richie; Flanagan, John J; Wustman, Brandon A; Barth, Jay; Barlow, Carrolee; Valenzano, Kenneth J; Lockhart, David J; Boudes, Pol; Johnson, Franklin K

The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidase

药理学伴侣异法戈明可增强戈谢病L444P突变型β-葡萄糖苷酶的活性。

Khanna, Richie; Benjamin, Elfrida R; Pellegrino, Lee; Schilling, Adriane; Rigat, Brigitte A; Soska, Rebecca; Nafar, Hadis; Ranes, Brian E; Feng, Jessie; Lun, Yi; Powe, Allan C; Palling, David J; Wustman, Brandon A; Schiffmann, Raphael; Mahuran, Don J; Lockhart, David J; Valenzano, Kenneth J